Promising enzyme therapy shows Long-Term safety for rare fabry disease
NCT ID NCT03614234
First seen Jan 26, 2026 · Last updated Jun 17, 2026 · Updated 22 times
Summary
This study looked at the long-term safety and effectiveness of a drug called pegunigalsidase alfa for adults with Fabry disease, a rare genetic condition. Participants received an infusion every four weeks for up to several years. The goal was to see if the drug is safe over time and helps control the disease.
Disclaimer
Read more
Show less
This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for FABRY DISEASE are added.
Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Locations
-
Addenbrooke's Hospital
Cambridge, CB2 0QQ, United Kingdom
-
Azienda Ospedaliera Universitaria "Federico II"
Naples, Via Pansini, 80131, Italy
-
Emory University School of Medicine
Atlanta, Georgia, 30307, United States
-
Fakultní poliklinika Všeobecné fakultní nemocnice v Praze
Prague, 128 08, Czechia
-
Helse Bergen HF Haukeland Universitetssykehus
Bergen, 5021, Norway
-
Infusion Associates
Grand Rapids, Michigan, 49525, United States
-
Medical Endocrinology PE 2132, Rigshospitalet
Copenhagen, 2100, Denmark
-
O & O Alpan
Fairfax, Virginia, 22030, United States
-
Renal Disease Research Institute, LLC
Dallas, Texas, 75235, United States
-
The Royal Free Hospital
London, United Kingdom
-
UAB Medicine
Birmingham, Alabama, 35233, United States
-
UZ Antwerpen
Edegem, 2650, Belgium
-
University of Iowa Hospitals and Clinica
Iowa City, Iowa, 52242, United States
-
University of Utah Hospitals & Clinics
Salt Lake City, Utah, 84112, United States
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.